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Article

Drug repositioning as a therapeutic strategy for neurodegenerations associated with OPA1 mutations.

Human molecular genetics - 21 Jan 2021

Aleo Serena J, Del Dotto Valentina, Fogazza Mario, Maresca Alessandra, Lodi Tiziana, Goffrini Paola, Ghelli Anna, Rugolo Michela, Carelli Valerio, Baruffini Enrico, Zanna Claudia

Abstract excerpt

OPA1 mutations are the major cause of dominant optic atrophy (DOA) and the syndromic form DOA plus, pathologies for which there is no established cure. We used a 'drug repurposing' approach to identify FDA-approved molecules able to rescue the mitochondrial dysfunctions induced by OPA1 mutations. We screened two different chemical libraries by using two yeast strains carrying the mgm1I322M and the chim3P646L...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 33231680
DOI: 10.1093/hmg/ddaa244
PMCID: PMC7823107

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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